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Therapeutic Oligonucleotides Targeting Liver Disease: TTR Amyloidosis
The liver has become an increasingly interesting target for oligonucleotide therapy. Mutations of the gene encoding transthyretin (TTR), expressed in vast amounts by the liver, result in a complex degenerative disease, termed familial amyloid polyneuropathy (FAP). Misfolded variants of TTR are linke...
Autores principales: | , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2015
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6332041/ https://www.ncbi.nlm.nih.gov/pubmed/26437390 http://dx.doi.org/10.3390/molecules201017944 |